The Clinibook
588 Pages
English

The Clinibook

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Description

This book provides a 2012 reference for state-of-the-art gene transfer technology and the different aspects of its clinical translation with a focus on European-based initiatives. As examples of successful outcomes, recent clinical trials are presented together with Ethical, Safety and Regulatory issues, which are discussed. The broad range of various technologies is covered whether addressing direct in vivo gene transfer like with AAV, Adeno or non-viral vectors or ex-vivo genetically engineered cells including induced pluripotent stem cells (iPS) with integrating vectors such as retrovirus, lentivirus or transposon-derived systems. The critical path to clinical implementation is covered in the second part describing currently available tools - such as molecular imaging, ex-vivo organ cultures and high-throughput technologies used for evaluation of criteria towards a go-or-no go decision to move to the clinic; in addition, utmost salient biosafety and immunotoxicology aspects are discussed.
This book is ideal for postgraduates, undergraduates, scientists, clinicians, regulators and patients' advocacy groups looking for states-of-the-art information as well as emerging prospects - including gene targeting and homologous recombination - in gene transfer intended for clinical translation.
Dr Odile Cohen-Haguenauer is the Coordinator of Clingene-NoE, the European Network for the Advancement of Clinical Gene Transfer and Therapy.

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Published by
Published 03 May 2013
Reads 78
EAN13 9782842542375
License: All rights reserved
Language English

Legal information: rental price per page €. This information is given for information only in accordance with current legislation.

The CliniBook
CLINICAL GENE TRANSFER STATE OF THE ART Edited by OD IL ECO H E N-HAG U E N A U E R
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The CliniBook
CLINICAL GENE TRANSFER: STATE-OF-THE-ART
Édîtîons EDK/Groupe EDP Scîences 25, rue Davîel 75013 Parîs, France Tél. : 01 58 10 19 05 Fax : 01 43 29 32 62 edk@edk.fr www.edk.fr
EDP Scîences 17, avenue du Hoggar PA de Courtabœuf 91944 Les Ulîs Cedex A, France www.edpscîences.org
© Édîtîons EDK, Parîs, 2012 ISBN : 978-2-8425-4171-2
Il est înterdît de reproduîte întégralement ou partîellement le présent ouvrage – loî du 11 mars 1957 – sans autorîsatîon de l’édîteur ou du centre Françaîs d’exploîtatîon du Droît de copîe (CFC), 20, rue des Grands-augustîns, 75006 Parîs, France.
TheCliniBook
CLINICAL GENE TRANSFER: STATE-OF-THE-ART
Edited by ODILECOHEN-HAGUENAUER
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« Hope lîes în dreams, în îmagînatîon and în the courage of those who dare to make dreams înto realîty » Jonas Salk, 1914-1995
« The joys of scîence lîe în the work îtself; the ultîmate reward beîng the progress of mankînd » Rosalînd Elsîe Franklîn, 1920-1958
« The utmost lîmîtatîons în scîence and dîscovery are not at the frontîers and complexîty of research but în people’s mînds: ît’s all about keepîng the faîth … »
à Julîen, Antoîne et Paul
CliniGene Partners and Boards
CLINIGENEACTINGPARTNERS
• Academic partners
ALESSANDRO AIUTI (and EUGENIO MONTINI),FCSR-TIGET, San Raffaele Telethon Instîtute for Gene Therapy, Mîlan, Italy; Medîcal Genetîcs, Department of Pedîatrîcs, “Federîco II” Unîversîty, Naples, Italy. ROBIN R. ALI,Instîtute of Opthalmology, UCL, London, England. ALBERTO AURICCHIO,Telethon Instîtute of Genetîcs and Medîcîne, FTELE-IGM, Napolî, Italy. SÉGOLÈNE AYMÉ,Orphanet, Inserm SC11, Plate-forme Maladîes Rares, Parîs, France. 1 CHRISTOPHER BAUM (and TONI CATHOMEN ),Medîzînîsche Hochschule Hannover, MHH, Hannover, Germany. FATIMA BOSCH,CBATEG-UAB, Unîversîtat Autonòma de Barcelona, Barcelona, Spaîn. JAN BUBENIK - MILAN REINIS,Instîtute of Molecular Genetîcs, IMG CAS, Prague, Czech Republîc. NATHALIE CARTIER,Inserm UMR 745, Unîversîty Parîs-Descartes, Sorbonne Parîs-Cîté, Parîs, France. KLAUS CICHUTEK (and MATTHIAS SCHWEIZER),Paul-Ehrlîch-Instîtut, PEI, Langen, Germany. MANUEL J.T. CARRONDO (and PEDRO E. CRUZ),Instîtuto de Bîologîa Experîmental e Tecnologîca, IBET-ITBQ, Oeîras, Portugal. 2 NICOLE DÉGLON,MIRCEN, Commîssarîat à l’Énergîe Atomîque-CEA, Saclay, France. GEORGES DICKSON (and RAFAEL J. YÁÑEZ-MUÑOZ),School of Bîologîcal Scîences, Royal Holloway-Unîversîty of London (RHUL), Egham, Surrey, Unîted Kîngdom MARK FEDERSPIEL - STEPHEN J. RUSSELL,Gene Therapy Program, Mayo Clînîc College of Medîcîne, Rochester, USA. GÖSTA GAHRTON,Department of Medîcîne, Karolînska Instîtutet-KI, Stockholm, Sweden. BERND GÄNSBACHER,Instîtute of Experîmental Oncology and Therapy Research, Klînîkum rechts der Isar der Technîschen Unîversîtät München, Munîch, Germany. MAURO GIACCA,Internatîonal Centre for Genetîc Engîneerîng and Bîotechnology, 3 ICGEB, Trîeste, Italy.
1 Now at Laboratory of Cell and Gene Therapy, Unîversîty Medîcal Center Freîburg, Engesserstr. 4, D-79108 Freîburg, Germany. 2 Now at Laboratory of Cellular and Molecular Neurotherapîes, Lausanne Unîversîty Medîcal School (CHUV), Lausanne, Swîtzerland. 3 A ClînîGene-SAB member untîl 2010.
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